The US Food and Drug Administration (FDA) has granted Orphan Drug Designation for the group’s cannabidiol (CBD) for the treatment of the rare genetic disorder, Tuberous Sclerosis Complex (TSC).
The drug is the third orphan indication that the group is targeting within it Epidiolec clinical development program. Earlier this month the group commenced phase III clinical trials for the CBD treatment as a therapy to treat seizures in combination with other TSC treatments.
A common symptom of TSC is epilepsy, which occurs in around 80-90% of sufferers and is the leading cause of morbidity and mortality.
The Epidiolec program includes a further four phase III pivotal trials in Dravet syndrome and Lennox-Gastaut syndrome, both rare and fatal forms of childhood-onset epilepsy.
The FDA Orphan Drug Designation entitles GW Pharmaceuticals to a seven-year exclusive market period.
Shares were up almost 2% on AIM this morning to 515p per share. On NASDAQ, GW shares last traded at $86.23 up by around 4%.